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诱导性多能干细胞疾病模型的应用价值

白雪   

  1. 天津市天津医院
  • 收稿日期:2013-07-04 修回日期:2013-10-23 出版日期:2014-04-15 发布日期:2014-04-15
  • 通讯作者: 白雪

The Application of the Induced Pluripotent Stem Cells Disease Modeling

  • Received:2013-07-04 Revised:2013-10-23 Published:2014-04-15 Online:2014-04-15

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摘要：

【摘要】诱导性多能干细胞（iPSC）技术为人类在体外研究疾病奠定了基础，利用该技术建立的iPSC疾病模型，已在众多疾病的研究中取得了重要进展。特别对于一些特异性遗传性疾病相对容易模拟，更适于采用iPSC疾病模型进行研究。利用iPSC技术可直接从患者身上获得所需的研究材料，不受伦理道德的限制，细胞来源丰富。所获得的疾病iPSC及其分化的细胞已被广泛用于病理机制研究、药物筛选及安全性检验、细胞治疗研究等。将来有望利用基因修复技术矫正患者致病基因，获得所需的靶细胞用于患者相关疾病的治疗。由于重编程过程造成细胞的改变、缺乏完善的分化实验方案、遗传背景差异等，使iPSC疾病模型存在局限性，采用更为先进的非整合性重编程技术、改善培养条件、执行严格的质量控制、建立iPSC疾病模型系统标准，将有助于解决这些问题。

关键词: 诱导性多能干细胞, 疾病模型, 病理机制, 药物筛选, 细胞治疗

Abstract: [Abstract] Induced pluripotent stem cells (iPSC) technology play an important role in studying diseases in vitro. iPSC disease model was based on the iPSC technology, and has achieved important results in many diseases. For those spe? cific genetic diseases that are relatively easy to simulate, iPSC disease model is especially useful. This technology can direct? ly obtained required material from patients, which is free from ethical constraints and rich in cell sources . iPSC disease model as widely used in studying disease mechanism, drug screening, safety inspection, cell therapy, and so on. It is expect? ed to be used in gene correction, gene repair and to obtain the required target cells for the treatment. Cells modification caused by reprogram, lacking of optimum experimental scheme for cell differentiation and difference in genetic backgrounde limit the application of iPSC disease model. More advanced nonintegrated programming technology, improvement of culture conditions, stricter quality control and setting up the standard for iPSC disease model will help to solve these problems.

Key words: Induced pluripotent stem cells, disease models, pathological mechanisms, drug screening, cell therapy